Public Perspectives on Multi-Cancer Early Detection: A Qualitative Study.

BACKGROUND: Multi-cancer early detection tests (MCEDs) have the potential to identify over 50 types of cancer from a blood sample, possibly transforming cancer screening paradigms. Studies on the safety and effectiveness of MCEDs are underway, but there is a paucity of research exploring public views on MCEDs. We sought to explore public perspectives and understanding on the use of MCEDs in patient care. METHODS: We conducted a cross-sectional, qualitative study using one-on-one, semi-structured interviews. Residents of the United States aged 45-70 years old were recruited through a survey panel and purposively sampled to maximize racial diversity. Interviews explored understanding of MCEDs and perspectives on their use. Interviews were analyzed using thematic analysis with deductive coding and semi-quantification. RESULTS: Among 27 participants, mean age was 62 years (range 48-70) and most (63%) were non-white. Most participants had completed at least one cancer screening (89%). Participants had a positive impression of MCEDs (85%) and found the concept easy to understand (88%). They were enthusiastic about the convenience of MCEDs (30%) and thought they would improve "cancer outcomes" by looking for multiple cancers (70%) and facilitating early detection (33%). Participants emphasized the need to balance these benefits against potential harms, including inaccuracy (96%), cost (92%), test-related anxiety (56%), and lack of evidence of effectiveness (22%). Participants favored that MCEDs be delivered in primary care (93%). Participants worried that the potential benefits of MCEDs might not be equitably distributed (44%). CONCLUSIONS: Members of the US public in this study expressed an interest in using MCEDs but had concerns regarding cost, accuracy, and potential inequitable access to the tests. Findings suggest that MCEDs that are found to be safe and effective will be acceptable to patients as a part of primary care, and underscore public interest in improving this technology.

Using best-worst scaling to inform policy decisions in Africa: a literature review.

BACKGROUND: Stakeholder engagement in policy decision-making is critical to inform required trade-offs, especially in low-and-middle income settings, such as many African countries. Discrete-choice experiments are now commonly used to engage stakeholders in policy decisions, but other methods such as best-worst scaling (BWS), a theory-driven prioritization technique, could be equally important. We sought to document and explore applications of BWS to assess stakeholder priorities in the African context to bring attention to BWS as a method and to assess how and why it is being used to inform policy. METHODS: We conducted a literature review of published applications of BWS for prioritization in Africa. RESULTS: Our study identified 35 studies, with the majority published in the past four years. BWS has most commonly been used in agriculture (43%) and health (34%), although its broad applicability is demonstrated through use in fields influencing social and economic determinants of health, including business, environment, and transportation. Published studies from eastern, western, southern, and northern Africa include a broad range of sample sizes, design choices, and analytical approaches. Most studies are of high quality and high policy relevance. Several studies cited benefits of using BWS, with many of those citing potential limitations rather than observed limitations in their study. CONCLUSIONS: Growing use of the method across the African continent demonstrates its feasibility and utility, recommending it for consideration among researchers, program implementers, policy makers, and funders when conducting preference research to influence policy and improve health systems. REGISTRATION: The review was registered on PROSPERO (CRD42020209745).

Pretesting Discrete-Choice Experiments: A Guide for Researchers.

Discrete-choice experiments (DCEs) are a frequently used method to explore the preferences of patients and other decision-makers in health. Pretesting is an essential stage in the design of a high-quality choice experiment and involves engaging with representatives of the target population to improve the readability, presentation, and structure of the preference instrument. The goal of pretesting in DCEs is to improve the validity, reliability, and relevance of the survey, while decreasing sources of bias, burden, and error associated with preference elicitation, data collection, and interpretation of the data. Despite its value to inform DCE design, pretesting lacks documented good practices or clearly reported applied examples. The purpose of this paper is: (1) to define pretesting and describe the pretesting process specifically in the context of a DCE, (2) to present a practical guide and pretesting interview discussion template for researchers looking to conduct a rigorous pretest of a DCE, and (3) to provide an illustrative example of how these resources were operationalized to inform the design of a complex DCE aimed at eliciting tradeoffs between personal privacy and societal benefit in the context of a police method known as investigative genetic genealogy (IGG).

Assessing the Benefits and Harms Associated with Early Diagnosis from the Perspective of Parents with Multiple Children Diagnosed with Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disorder diagnosed in childhood. Limited newborn screening in the US often delays diagnosis. With multiple FDA-approved therapies, early diagnosis is crucial for timely treatment but may entail other benefits and harms. Using a community-based survey, we explored how parents of siblings with DMD perceived early diagnosis of one child due to a prior child's diagnosis. We assessed parents' viewpoints across domains including diagnostic journey, treatment initiatives, service access, preparedness, parenting, emotional impact, and caregiving experience. We analyzed closed-ended responses on a -1.0 to +1.0 scale to measure the degree of harm or benefit parents perceived and analyzed open-ended responses thematically. A total of 45 parents completed the survey, with an average age of 43.5 years and 20.0% identifying as non-white. Younger siblings were diagnosed 2 years earlier on average (p < 0.001). Overall, parents viewed early diagnosis positively (mean: 0.39), particularly regarding school preparedness (+0.79), support services (+0.78), treatment evaluation (+0.68), and avoiding diagnostic odyssey (+0.67). Increased worry was a common downside (-0.40). Open-ended responses highlighted improved outlook and health management alongside heightened emotional distress and treatment burdens. These findings address gaps in the evidence by documenting the effectiveness of early screening and diagnosis of DMD using sibling data.

Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals.

BACKGROUND: Despite the increasing availability of clinical trials in Duchenne muscular dystrophy, racial/ethnic minorities and other populations facing health disparities remain underrepresented in clinical trials evaluating products for Duchenne. We sought to understand the barriers faced by Hispanic/Latino families specifically and underrepresented groups more generally to clinical trial participation in Duchenne. METHODS: We engaged two participant groups: Hispanic/Latino caregivers of children with Duchenne in the US, including Puerto Rico, and health professionals within the broader US Duchenne community. Caregiver interviews explored attitudes towards and experiences with clinical trials, while professional interviews explored barriers to clinical trial participation among socio-demographically underrepresented families (e.g., low income, rural, racial/ethnic minority, etc.). Interviews were analyzed aggregately and using a thematic analysis approach. An advisory group was engaged throughout the course of the study to inform design, conduct, and interpretation of findings generated from interviews. RESULTS: Thirty interviews were conducted, including with 12 Hispanic/Latina caregivers and 18 professionals. We identified barriers to clinical trial participation at various stages of the enrollment process. In the initial identification of patients, barriers included lack of awareness about trials and clinical trial locations at clinics that were less likely to serve diverse patients. In the prescreening process, barriers included ineligibility, anticipated non-compliance in clinical trial protocols, and language discrimination. In screening, barriers included concerns about characteristics of the trial, as well as mistrust/lack of trust. In consent and recruitment, barriers included lack of timely decision support, logistical factors (distance, time, money), and lack of translated study materials. CONCLUSIONS: Numerous barriers hinder participation in Duchenne clinical trials for Hispanic/Latino families and other populations experiencing health disparities. Addressing these barriers necessitates interventions across multiple stages of the clinical trial enrollment process. Recommendations to enhance participation opportunities include developing clinical trial decision support tools, translating prominent clinical trials educational resources such as ClinicalTrials.gov, fostering trusting family-provider relationships, engaging families in clinical trial design, and establishing ethical guidelines for pre-screening potentially non-compliant patients.

Willingness and preferences for long-acting injectable PrEP among US men who have sex with men: a discrete choice experiment.

INTRODUCTION: Cabotegravir long-acting injectable HIV pre-exposure prophylaxis (LA-PrEP) was shown to be safe and effective in multiple clinical trials. Increasing uptake and persistence among populations with elevated risk for HIV acquisition, especially among men who have sex with men (MSM), is critical to HIV prevention. OBJECTIVE: This analysis aims to understand potential users' preferences for LA-PrEP, with audience segmentation. DESIGN: Willingness to use and preferences for LA-PrEP were measured in HIV-negative, sexually active MSM in the 2020 American Men's Internet Survey. Respondents answered a discrete choice experiment with paired profiles of hypothetical LA-PrEP characteristics with an opt-out option (no LA-PrEP). Conditional and mixed logit models were run; the final model was a dummy-coded mixed logit that interacted with the opt-out. SETTING: US national online sample. RESULTS: Among 2506 MSM respondents, most (75%) indicated a willingness to use LA-PrEP versus daily oral PrEP versus no PrEP. Respondents were averse to side effects and increasing costs and preferred increasing levels of protection. Respondents preferred a 2-hour time to obtain LA-PrEP vs 1 hour, with a strong aversion to 3 hours. Overall, there was an aversion to opting out of LA-PrEP, with variations: those with only one partner, no/other insurance or who were Black, Indigenous or People of Colour were significantly less likely to prefer LA-PrEP, while those who were Hispanic/Latino, college educated and <40 years significantly preferred LA-PrEP. CONCLUSIONS: A large proportion of MSM expressed a preference for LA-PrEP over daily oral pills. Most respondents chose LA-PrEP regardless of cost, clinic time, side effects or protection level; however, preferences varied by sociodemographics. These varied groups likely require tailored intervention strategies to achieve maximum LA-PrEP uptake and persistence.